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(11)

EP 4 037 704 A1

(12)

(43) Date of publication: 10.08.2022 Bulletin 2022/32 (21) Application number: 20870591.3 (22) Date of filing: 02.10.2020

(51) International Patent Classification (IPC):  A61K 38/17(2006.01) A61P 9/10(2006.01) A61P 7/02(2006.01) (52) Cooperative Patent Classification (CPC): A61P 7/02; A61P 9/10; A61K 38/4886; C12Y 304/24082; A61K 45/06 (86) International application number: PCT/US2020/053901 (87) International publication number: WO 2021/067668 (08.04.2021 Gazette 2021/14)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA ME Designated Validation States: KH MA MD TN (30) Priority: 04.10.2019 US 201962911019 P (71) Applicant: The Children's Medical Center Corporation Boston, Massachusetts 02115 (US)	(72) Inventors: WAGNER, Denisa D. Dover, Massachusetts 02030 (US) WONG, Siu Ling Singapore Level 10, 11 Manndalay Road Singapore 308232 (SG) (74) Representative: Keltie LLP  No. 1 London Bridge London SE1 9BA London SE1 9BA (GB)